Pharming Technologies B.V.

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Trial	Conditions	Status	Phase	Updated	NCT ID
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease	Nutritional and Metabolic Diseases, inborn mitochondrial metabolism disorder, metabolic disease	Recruiting	Phase 2	2026-09-16	NCT07514338
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation	PIDs Linked to PI3K	Active, not recruiting	Phase 2	2026-09-16	NCT06990529
Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease	Muscle Weakness, Neurologic Manifestations, Neuromuscular Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, diabetes mellitus, endocrine system disorder, glucose metabolism disease, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Recruiting	Phase 2	2026-06-26	NCT05650229
Pediatric Patients Aged 1 to 6 Years With APDS	activated PI3K-delta syndrome	Active, not recruiting	Phase 3	2026-06-15	NCT05693129
Pediatric Patients Aged 4 to 11 Years With APDS	activated PI3K-delta syndrome	Active, not recruiting	Phase 3	2026-05-06	NCT05438407
Leniolisib for Immune Dysregulation in PIDs	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Primary Immunodeficiency Diseases, hereditary disease, immune system disorder, inborn error of immunity	Active, not recruiting	Phase 2	2026-02-13	NCT06549114

Clinical trials